Global Alzheimer s Platform. Luc Truyen, ltruyen8@its.jnj.com George Vradenburg, vradenburg@aol.com Debra Lappin, debra.lappin@faegrebd.

Transcription

1 Global Alzheimer s Platform 2015 Luc Truyen, ltruyen8@its.jnj.com George Vradenburg, vradenburg@aol.com Debra Lappin, debra.lappin@faegrebd.com 1

2 Presentation 1. Today s Reality 2. Global Momentum 3. GAP Vision and Progress 4. Request & Discussion 2

3 Today s clinical trial reality in AD 3

4 Today s clinical trial reality in AD Too late? Fire and Forget trials Diagnosis? Intervention? 4

5 Momentum for Global Response to Dementia Key Events: 2012: Formation of the Global CEO Initiative on Alzheimer s 2013: Dementia Summit and follow-on events: London/Finance, Ottawa/PPP, Toronto/Big Data, Tokyo/Care, Lausanne/Regulation 2014: Formation of GAP: Drives global integration of clinical trials platform for secondary prevention of Alzheimer s and Dementia: EU, Canada, US 2014: OECD meeting on global harmonization of regulatory path for AD 2015: GAP alignment with IMI-EPAD; WHO; World Dementia Conference Global Funding: Europe/IMI-EPAD: Committed public investment of ~E25M across 5 yrs (+ IMI EMIF and AETIONOMY) Canada/CCNA: Application for ~$12.5M Canadian public investment over 5 yrs US/GAP: Seeking minimum of $25M public investment over 5 yrs to be complemented with private funding and leadership 5

6 GAP Vision The overall vision is to establish a global standing trial-ready platform which is projected to reduce clinical testing cycle times by two years or more and achieve greater efficiency and uniformity in trial populations through large, well-characterized trial-ready cohorts, certified clinical trial sites, and an adaptive proof of concept trial mechanism. This platform will enable the delivery of efficient and effective proof of concept and confirmatory trials and ultimately the more rapid delivery of effective therapies to patients or those at risk. 6

7 The Pieces of the Puzzle coming together in global alignment Better understand AD and its evolution Identify new targets and validate them -> new assets AD Budget: 5 years ($129.5 Million Total Project Funding) Optimized learning and confirming An integrated approach accelerates path to patients 7

8 Clinical Landscape Today Current Global Assets a Foundation to Build On: US: Long-standing database/cohorts (ADRC/NACC); Standing sites (ADCS, DIAN) Global Trials as essential platform Standardized methods (imaging, biochemical biomarkers, cognitive assessments, analytic approaches, quality methods (monitoring, site metrics)) Refined diagnostic criteria and tools to enable earlier interventions Current Bottlenecks: No existing National or International IRB Do not have full global harmonization No ongoing support to maintain trial site quality standards (raters, data management, imaging, etc.) between trials No trial-ready cohort No framework for primary prevention or combination trials No common platform for cognitive/biomarker trials 8

9 Value Proposition: Speeding Clinical Trials Clinical Trial Infrastructure Offer network publicly -and industry-sponsored POC or Confirmatory trials Master Services Agreement Site rater certification National IRB Site Accreditation Prospective Readiness Cohort Study No lag time for recruitment Significantly lower screen failure Diagnostic confidence Quality run in data Test drive clinical end-points Research and validate biomarkers Amortize costs Biomarker-rich Cognitive Endpoint Trials Offer platform for publicly- and industry-sponsored POC or Confirmatory trials Decrease risk/time; Improve decisions for Ph 3 Develop protocol for Ph 2/3 global POC (EPAD, CPAD, etc.) Public-Private Partnership essential to foster innovative trials that would not be done by industry alone (e.g., novel cognitive/biomarker endpoints, combination trials, primary prevention, etc.); Data made available to field. 9

10 AD Clinical Research Value Proposition Current Bottlenecks: Difficulty identifying and enrolling trial participants Current recruitment: pts/site/month High screen failure rate Need for 100 s of sites globally complexity in training, monitoring of personnel, data management Infrastructure is rebuilt trial-by-trial Significant monetary investment Increasing demand on clinical trial infrastructure Quality of data GAP Opportunity: 10

11 GAP Opportunity: A Look at the AD Clinical Pipeline Robust clinical pipelines exist Public investment (ADNI, AMP) in AD will spur development of novel therapies Ph 3 clinical success will drive significant increase in discovery/ development efforts Clinical trial demand requires more reliable and efficient trial infrastructure addressed by GAP AD Modeling Assummptions 25 molecules enter Ph 1/yr P(TS) Duration Phase 1 70% 2 Phase 2 40% 3 Phase 3 n/a 4 Pipeline Data Sources: B Munos, J&J AD, Citeline AD 11

12 GAP Work Streams: Enhancing Current AD Ecosystem GAP Builds On Current NIH/NIA Initiatives(*) and AD Ecosystem and Drives Unique Value Across POC and Confirmatory Clinical Trials Recruitment Channels Patient Coordinating Centers (eg, ADRCs, ADCS, CTSA, PCORI) Online Patient Registries (eg, BHR) Specialized Big Data Co s (eg, Healthcare Informatics, CROs) PCP Networks and/ Health System Providers (Mayo, Banner) 2. GAP Federated Registry GAP Registry 3. Data Harmonization Prospective Readiness Cohort Study Global POC Study Arm 1 Global POC Study Arm 2 Ph 3 Clinical Trial 1 1. Diversified Recruitment Channels 4. Longitudinal Subject Cohort Data and Clinical Trial Ready Characterization Ph 3 Clinical Trial 2 (*) ADCS, ADRC s, NACC 5. Pre-certified Sites Cohort Study, POC, and Confirmatory CONFIDENTIAL: These slides and the materials contained within are confidential and should not be shared without permission of MTR. Please contact via mropacki@its.jnj.com 12

13 Trial Innovation: Model Proof of Concept Study GAP RFA will fund key academic engagement with POC protocol and trial definition and design. Biomarkers/Cognition Drivers of Adaptation Cognitive Proof of Concept Refer IMI-EPAD 13

14 GAP: Implementation Plan 3Q2015 4Q GAP Registry Clinical Trial Site Clinical Trial Site 1. Build GAP Registry off BHR backbone; 2. Certify select sites Prospective Readiness Cohort Study Site Cohort Study Prospective Readiness Cohort Study Site 3. Launch Cohort Study with limited number of certified cohort study sites 2015: Launch GAP Register: add ~2000 subjects Launch Cohort Study: add ~200 participants Certify ~25 sites including cohort study sites ~$5M Clinical Trial Site Clinical Trial Site Clinical Trial Site 4. Expand Cohort Study Sites; 5. Certify GAP network sites for global POC and Confirmatory Studies; 6. Launch global POC study 2016: Expand Register: ~4000 subjects Expand Cohort Study ~400 participants Certify up to 100 sites ~$11M 7. Expand GAP network sites for global POC and Confirmatory Studies : Expand Register: ~6000 subjects Expand Cohort Study: ~1200 participants Maintain certification on up to 100 sites 2017 ~$14M; 2018 ~$18M 14